Independent validation for Asokan Delivery Team: Evolving High Potency AAV Vectors for Neuromuscular Genome Editing. |
cc47-SaCas9, Male |
Fluorescent imaging for tdTomato+ cells |
pancreas |
Editing Efficiency |
15000001261 |
|
SauCas9 |
|
|
Ai9 SaCas9 Guide A; Ai9 SaCas9 Guide B |
AAVcc47-SaCas9-Ai9; |
|
Mouse |
B6.Cg-Gt(ROSA)26Sor ^ tm9(CAG-tdTomato)Hze/J |
|
intravenous (tail vein) |
liver and heart |
1.0x10^14 vg copies/kg (vg/kg) (2.0x10^12/20g mouse) |
once |
|
200 microliters |
4 weeks |
AAV vector encoded |
|
|
|
Measured Values |
Samples Size: | 6 |
|
Mean |
3 No red cells; 1 One red cell; 1 Two red cells; 1 A few red cells  |
1 |
No red cells  |
2 |
No red cells  |
3 |
Two red cells  |
4 |
No red cells  |
5 |
One red cell  |
6 |
A few red cells  |